NCT00008450

Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant

Induction of Mixed Hematopoietic Chimerism in Patients With Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Fred Hutchinson Cancer Center · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This pilot clinical trial studies total-body irradiation followed by cyclosporine and mycophenolate mofetil in treating patients with severe combined immunodeficiency (SCID) undergoing donor bone marrow transplant. Giving total-body irradiation (TBI) before a donor bone marrow transplant using stem cells that closely match the patient's stem cells, helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may mix with the patient's immune cells and help destroy any remaining abnormal cells. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after the transplant may stop this from happening.

Detailed description

PRIMARY OBJECTIVES: I. To safely establish partial lymphoid chimerism (1-95% donor cluster of differentiation \[CD\]3+ cells) using a non-lethal conditioning regimen in patients with severe combined immunodeficiency syndrome. II. To define the kinetics of immune reconstitution following a non-lethal conditioning regimen in patients with immunodeficiency diseases. OUTLINE: Patients receive cyclosporine orally (PO) or intravenously (IV) on days -3 to 100 followed by a taper until day 180 and mycophenolate mofetil PO or IV on days 0-40 with a taper until day 96 in the absence of unacceptable toxicity. Unrelated donor recipients also undergo TBI on day 0. Patients undergo bone marrow transplant on day 0. After completion of study treatment, patients are followed up at 6 months and then yearly for 5 years.

Conditions

• Adenosine Deaminase Deficiency
• Autosomal Recessive Disorder
• Immune System Disorder
• Purine-Nucleoside Phosphorylase Deficiency
• Severe Combined Immunodeficiency
• Severe Combined Immunodeficiency With Absence of T and B Cells
• X-Linked Severe Combined Immunodeficiency

Interventions

Type	Name	Description
PROCEDURE	Allogeneic Bone Marrow Transplantation	Undergo allogeneic bone marrow transplant
DRUG	Cyclosporine	Given PO or IV
OTHER	Laboratory Biomarker Analysis	Correlative studies
DRUG	Mycophenolate Mofetil	Given PO or IV
PROCEDURE	Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation	Undergo nonmyeloablative allogeneic hematopoietic stem cell transplant
RADIATION	Total-Body Irradiation	Undergo TBI

Timeline

• Start date: 1997-08-11
• Primary completion: 2011-10-25
• Completion: 2018-12-26
• First posted: 2001-01-08
• Last updated: 2019-07-29

Locations

1 site across 1 country: United States